The Landscape of Cellular and Gene Therapy Products: Cost, Approvals, and Discontinuations
Notice bibliographique
Résumé
Background The past 10 years witnessed a significant increase in the approval of cellular and gene therapy products worldwide. The US Food and Drug Administration (FDA) approved 3 gene therapy products within the last 4 months of 2017. The objective of this study was to examine the approval characteristics, discontinuations and cost of all cellular and gene therapy products approved worldwide. Data and Methods We conducted an electronic search of approved cell and gene therapy products from the databases of the main drug regulatory agencies including the US Food and Drug Administration, the European Medicines Agency (EMA), the Korea Ministry of Food and Drug Safety (MFDS), and Japan’s Pharmaceuticals and Medical Devices Agency, China Food and Drug Administration, Ministry of Health and Social Development-Russia, Health Canada, and the Food and Drug Administration of the Philippines. We also searched the literature using MEDLINE/PubMed, Cochrane Library, Google Scholar and EMBASE databases. Cost information from the US was derived from the Reb Book (Truven Health Analytics). Costs from Europe were derived from Health Technology Assessments and from public sector, financial news and company’s web pages. Where available, we also obtained cost for products approved in other countries from public sector, financial news and company’s web pages. All cost data were converted to USD. Results There were 52 cell and gene therapy products marketed in the world as of September 2018. Of these, 39 (75.0%) were cell therapy medicinal products and 13 (25.0%) were gene therapy medicinal products. Of the approved products, 29 (74.3%) cell and 8 (61.5%) gene therapy products were first approved in the past 10 years (2008-present). Korea had the greatest number of approved cellular therapy-19 followed by US-12, Japan -4, Europe -3 and Canada -1. While Europe had the highest number of approved gene therapies - 5 followed by US - 3, China -2 and Korea, Russia and Philippines one each. Overall, 8 (66.7%) of the approved gene therapy products were granted orphan designation. Three products approved by the EMA were withdrawn or discontinued from the market. Of them, 2 remain currently marketed in the US. Cost of treatment from Gene therapy in US ranged from $447600 to $1,020,000 while in Europe the range was $370000 to $962890. Gene therapies outside US and Europe were relatively cheaper. Cell therapies in North America had a price range from $30,370-$200,000, European cell therapies ranged from $17,658-$105,000. Conclusion In the study period, more autologous cell therapy products were approved than allogenic agents. The cost of gene and cell therapy drugs is much higher in the EU and North America in comparison to Asia. Majority of the gene therapies received orphan designation by the regulatory authorities and had conditional approvals while less than a quarter of cellular therapies received the orphan designation. Most of the gene therapies are approved for rare diseases with smaller patient population, companies find it difficult to make profits which results in market withdrawal of the therapies.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,000 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule tête enseignante, pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».